Author: admin

In September 2015, I wrote about the U.S. government’s deliberative process to re-evaluate the risks and benefits associated with certain “gain-of-function” studies involving pathogens.  Such studies are undertaken to better understand how pathogens infect people and spread through the population. These studies help assess the risks associated with emerging pathogens, particularly those like new influenza strains that may have pandemic potential.

However, these studies can also generate pathogens with enhanced characteristics, such as pathogenicity (ability to cause disease) or transmissibility (ability to spread) in mammals.  Concerns have been raised about whether a modified laboratory pathogen could be accidentally or intentionally released from a lab, threatening local or even global populations.  Some have questioned whether such studies are worthwhile.

The National Science Advisory Board for Biosecurity (NSABB) has been examining the issue and on January 7 and 8, on the NIH campus, the Board will convene to discuss the risks and benefits, as well as ethical and policy issues.  To inform NSABB deliberations, NIH commissioned a thorough risk and benefit assessment and an ethical analysis—the results of both will be presented for the first time at the upcoming meeting.  NSABB will also begin discussing its preliminary findings and a potential path forward for funding and conducting gain-of-function studies in pathogens with pandemic potential.

The meeting is free and open to the public.  It will also be webcast and questions during the meeting can be submitted to the NSABB inbox at [email protected].  Written comments on the topic can also be submitted to NSABB at the same address. See here to register for the meeting and to learn more.

The gain-of-function debate comes at a time when we are having similar discussions about how to rapidly and responsibly advance new biomedical technologies to promote public health and well-being. It raises difficult policy questions about how to maximize the potential benefits of some areas of scientific research while mitigating potential risks.  I hope you can join us for what should be a very interesting meeting.

‘Twas the night before Christmas, and at NIH,

There were still policy issues, keeping us wonks awake.

The sIRB comments have been read with care,

Soon a final policy will be ready to share.

The Common Rule comments are still flowing in,

Thoughts on consent forms for biospecimens.

NSABB staff are busy, organizing fora to discuss,

The Gain of Function studies that have raised such a fuss.

And I at my computer, and staff at theirs, too,

Are pushing along the CT.gov final rule.

Precision medicine and chimeras are both front of mind,

Biosafety compliance is not far behind.

The new Strategic Plan raised a small clatter,

As everyone read it to find what really matters.

Data sharing policy, don’t forget about that,

Even as we update our disease burden stats.

The Biennial report is soon to emerge,

Full of good info – read it, we urge!

The RAC staff is ready, FR comments in hand,

To implement the streamlined process we planned.

Then what to our wondering eyes should appear,

But an omnibus bill for this fiscal year!

And as OSP Director, true policy geek,

I keep policy wheels rolling, week after week,

“Now, consent! Now, DURC! Now, gametes and eggs!

On NPRM! On agencies and regs!

To extramural and intramural, and all in between,

To new science policy in 2016!”

The Office of Science Policy (OSP) has added technology transfer and innovation policy to its scope of advice and support to the NIH Director on issues affecting the biomedical research community, the NIH, and the public.  As described previously, OSP participates in the development of new policies and programs in a variety of arenas that span the research continuum from basic laboratory science, through translational and clinical research, and from domestic to global public health.   With the addition of tech transfer policy to our portfolio, OSP staff now collect data and assess activities involving the commercialization of technologies based on NIH funded research.  Ongoing research programs invariably require the application of policies related to technology transfer. In addition, the advent of new technologies and collaboration paradigms raises questions and may merit application of new policies.

For example, in receiving a material from a third party, what level of restrictions, if any, is NIH willing to accept on its use, the publication of research results, and ownership of newly developed materials?  If a new inventive technology arises, will it be patented, what institution(s) owns the patent, and who has rights to use it for research or commercial use?  These are typically fact-specific questions made by NIH Institutes and Centers (ICs) and grantees.   However, policy guidance shapes where the lines are drawn and when.  Access to new technologies and creative collaborative arrangements may require creative applications of existing policies or the development of new ones.

While taking on a leading role within the Office of the Director, OSP works closely with the Office of Intramural Research (OIR), the Office of Extramural Research (OER), and ICs on policy issues within their respective domains.  In the past, technology transfer policy issues have included arrangements for the receipt and distribution of approved stem cell lines, patenting of genomic technologies, and the restrictions under which samples of pandemic flu strains would be distributed internationally to researchers and organization preparing vaccine seed strains.  The reorganization of technology transfer at NIH and the introduction of staff focused on technology transfer in OSP have led to the newly created Technology Transfer and Innovation Policy Division.   Among our areas of current interest: the evolving nature of complex prognostic tests requiring greater clinical development prior to commercialization and an analysis of drugs and biologics approved for precision medicine applications.

This staffing of expertise within OSP will benefit the OD and the NIH community in strengthening the management and oversight of policy functions and the programs that provide vital support to the NIH community on matters involving technology transfer and innovation policy.

Today, NIH is releasing a Guide Notice on Clarifying NIH’s Priorities in Health Economics, demonstrating the importance NIH places on supporting research that examines “how scarce resources are allocated among alternative uses for the care of sickness and the promotion, maintenance, and improvement of health, including the study of how health care and health-related services, their costs and benefits, and health itself are distributed among individuals and groups in society.”  In other words, how economic models and methods can be used to support NIH’s mission to use fundamental knowledge to enhance health, lengthen life, and reduce illness and disability.

Health economics can provide rigorous tools for answering important questions about how new medical innovations are adopted and deployed; about how patients and doctors can make the best informed choices for treatment and prevention; and about how different ways of delivering health care can affect the health of individuals and populations.   Answering these sorts of questions is vital to our work here at NIH, and as of 2015, NIH funding has supported the work of five Nobel Laureates in Economic Sciences who made great advancements in our understanding of health and society.  In fact, health economics is only one example of the type of behavioral and social science research funded by NIH that is absolutely central to our ability to prevent and treat disability and disease.

Today’s Guide Notice clearly demonstrates the importance that NIH places on supporting health economics research in which health outcomes and health-related behaviors are the primary focus, and the connection between the subject(s) of the study and improved understanding of health are clear and explicit.  Health economics research that makes a strong, explicit tie to health and health-related outcomes is central to the NIH mission, and NIH believes that such studies are a worthy investment of taxpayer funds.  It is also true that an economic analysis is often included as one piece of a larger study and that such analysis is often part of understanding the real-world consequences of health interventions. Studies where the primary focus of the research is not health economics, but include such analyses as a secondary aim, continue to be a valued part of the NIH portfolio.

Some topics and approaches which are not necessarily NIH-wide priorities may still be priorities for the missions of individual Institutes and Centers. Principal Investigators (PIs) and potential PIs for NIH research grants should consult with NIH program officers in Institutes and Centers appropriate to their proposed topic if they have questions about whether their work will fit program priorities.

The Notice also identifies study topics outside the NIH mission, which will not be funded by the agency. These topics, although potentially valuable areas of research, do not connect clearly to NIH’s mission or priorities related to the understanding of health, and therefore may be a better fit at other organizations and agencies. This underscores NIH’s strong commitment to responsible stewardship of the taxpayer dollars and to transparency in setting priorities for the agency.